Sökning: "gene therapy"

Visar resultat 16 - 20 av 696 avhandlingar innehållade orden gene therapy.

  1. 16. Precision mapping of gene expression and proteins in the brain using gene editing and barcoded viral vectors

    Författare :Martino Avallone; Molekylär neuromodulering; []
    Nyckelord :MEDICIN OCH HÄLSOVETENSKAP; MEDICAL AND HEALTH SCIENCES; Gene editing; CRISPR Cas9; Barcodes; AAV; PLA; gene therapy; Parkinson’s disease; single nuclei RNAseq; alpha-synuclein;

    Sammanfattning : The human brain is a masterpiece of intricate design and impeccable functionality. It serves as the ultimate command center for our thoughts, sensations, and actions, which define our very existence. LÄS MER

  2. 17. Towards Gene Therapy of Osteopetrosis

    Författare :Maria Askmyr; Avdelningen för molekylärmedicin och genterapi; []
    Nyckelord :MEDICIN OCH HÄLSOVETENSKAP; MEDICAL AND HEALTH SCIENCES; extracellular fluids; Haematology; oc oc mice; osteoclast; hematopoietic stem cells; Hematologi; extracellulära vätskor;

    Sammanfattning : The goal in this thesis is development of gene therapy for malignant infantile osteopetrosis (IMO), a rare but severe genetic bone disease. The concept of osteopetrosis implies dysfunction or lack of osteoclasts, the bone resorbing cells in our body, resulting in failure of normal bone breakdown. LÄS MER

  3. 18. Gene therapy in a new model of delayed wound healing

    Författare :Patrik Velander; Kirurgi; []
    Nyckelord :MEDICIN OCH HÄLSOVETENSKAP; MEDICAL AND HEALTH SCIENCES; IGF-1; Gene therapy; Diabetes; Animal model; EGF;

    Sammanfattning : Diabetes wounds result in significant morbidity, prolonged hospitalization and huge health care expenses. As of yet, no optimal animal models of diabetic wound healing exist to study potential beneficial therapies. Porcine wound healing is very similar to human wound healing. LÄS MER

  4. 19. Vector development for suicide gene therapy

    Författare :Alar Aints; Karolinska Institutet; Karolinska Institutet; []
    Nyckelord :Suicide gene therapy; VP22; selection markers; Ouabain resistance gene; GvHD; retroviral gene transfer;

    Sammanfattning : Gene therapy is used to treat conditions that arise from errors in the genetic makeup of cells either congenital diseases resulting from a deletion or mutation in a gene or malignant diseases where genetic regulation mechanisms have been deranged. Suicide gene therapy is one of several gene therapeutic approaches to treat cancer. LÄS MER

  5. 20. Osteogenesis Imperfecta : Genetic and Therapeutic Studies

    Författare :Katarina Lindahl; Östen Ljunggren; Andreas Kindmark; Carl-Johan Rubin; David Sillence; Uppsala universitet; []
    Nyckelord :MEDICIN OCH HÄLSOVETENSKAP; MEDICAL AND HEALTH SCIENCES; OI; BMD; Genotype; Phenotype; Pharmaco-genetics; Bisphosphonate; Therapy; Gene-therapy; Mutation; Collagen; Collagen type I; Allele-specific silencing; siRNA; RNAi; COL1A1; COL1A2; Stroke; C-propeptide; Mineralization; Heterozygous disadvantage; Genetics; Genetik; Medicin; Medicine; Medical Genetics; Medicinsk genetik;

    Sammanfattning : Osteogenesis imperfecta (OI) is a heterogeneous disease of connective tissue, the cardinal symptom being fractures and severity ranging from mild to lethal. Dominant mutations in collagen I, encoded by COL1A1 and COL1A2, cause >90% of cases. LÄS MER